Policy
The FDA’s drug review process can often be “unpredictable,” and review teams typically “differ greatly” in what they ask of drug sponsors, Sen. Bill Cassidy said.
FEATURED STORIES
Bristol Myers Squibb, GSK and Merck are contributing drug ingredients as part of their deals with the White House but are keeping many of the terms of their agreements private.
Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis by the Rare Disease Company Coalition shows. That could mean $4 billion in missed revenue for already cash-strapped biotechs.
The FDA’s rare pediatric disease priority review voucher program missed reauthorization at the last minute in 2024; advocates have been fighting to get it back ever since.
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The third quarter of 2022 saw continued job cuts, a shift in focus away from COVID-19, an unexpected boost in the Alzheimer’s research space and increased scrutiny of accelerated approvals.
The FDA’s approval of Amylyx’s Relyvrio has ignited the growing momentum in the ALS space. BioSpace spoke with Amylyx co-CEOs Justin Klee and Josh Cohen and leading ALS researcher Dr. Merit Cudkowicz.
Theratechnologies’ Trogarzo for HIV, Alnylam’s Lumasiran for advanced primary hyperoxaluria, scPharmaceuticals’ Furoscix for heart failure and more. Here’s a look at the FDA’s October calendar.
The FDA had a busy week, approving drugs, greenlighting clinical trials and other regulatory activities for Immuneering, Amylyx, Sarepta, Sanofi, and Regeneron and more.
In its ongoing battle against counterfeit drug rings, Gilead Sciences announced it has identified two “kingpins” behind the scheme to sell illegitimate HIV medications.
The HHS Office of Inspector General found that of 278 accelerated approvals between 1992 and 2021, 104 had incomplete confirmatory trials, according to a report issued Thursday.
Two years after receiving a Complete Response Letter for its hemophilia gene therapy, BioMarin is heading back to the FDA with stronger and more robust data for potential approval.
PhaseBio Pharmaceuticals reported its collaboration partner, SFJ Pharmaceuticals Group, informed the company it would need to return the rights to its lead program.
The FDA ordered Avidity Biosciences to halt clinical trial enrollment after a patient suffered a “serious adverse event” testing the company’s candidate drug for myotonic dystrophy type 1.
The FDA has granted a rolling review to CRISPR Therapeutics and Vertex’s exa-cel, a potential one-time treatment for sickle cell disease and transfusion-dependent beta-thalassemia.